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Stem Cell Transplant for Sickle Cell Disease

Treatment Overview

Stem cell transplantation is a potential cure for sickle cell disease. Stem cells can be found in bone marrow. Bone marrow is the substance in the center of your bones that produces red blood cells. A person with sickle cell disease has bone marrow that produces red blood cells with defective hemoglobin S. But if that bone marrow is replaced with healthy bone marrow, a person's body may start to produce normal hemoglobin.

Before the transplant, bone marrow stem cells are taken from someone who has closely matching bone marrow, usually a healthy brother or sister. The child who has sickle cell disease is then treated with drugs that destroy his or her bone marrow cells. After that, the donated bone marrow stem cells are injected into a vein.

After the process is complete, the donor's bone marrow begins to replace the recipient's bone marrow. These new cells restore the immune system and make normal red blood cells.

Until recently this treatment was mostly done for children. This is because the risk of problems increases with a person's age. But new methods of doing a stem cell transplant are making it possible to successfully treat adults.

What To Expect

After stem cell transplant, recovery takes 1 to 2 months in the hospital. The child's natural defense system needs this time to start working again.

During recovery, doctors watch closely for signs that the immune system is rejecting the new bone marrow and for signs of infection. If a problem occurs, recovery can take longer or the transplant may fail.

Why It Is Done

Stem cell transplants offer a potential cure for a child's sickle cell disease. They are usually considered only for children younger than 16 who have:footnote 1

The risks of stem cell transplant become greater as a person gets older and/or develops damage to major organs. For these reasons, a bone marrow transplant is not a treatment option for most adults who have sickle cell disease. But research on bone marrow transplants in adults is ongoing.

How Well It Works

If successful, a bone marrow transplant can cure sickle cell disease. This treatment has been successful in about 85 out of 100 children who had transplants.footnote 2, footnote 3 But the risk of dying after a transplant is about 5%.footnote 4

Risks

Any bone marrow transplant is risky, because complications, such as severe infections and immune system problems, can occur.

If the recipient's natural defense (immune) system isn't weakened enough by the medicines given before the transplant, it can attack the new stem cells and cause the transplant to fail.

If the donor's stem cells don't match the recipient's closely enough, the donor's new immune system cells may attack certain organs in the recipient (graft-versus-host disease).

A person may be unable to have children (infertile) after a bone marrow transplant. But new methods of doing this procedure are reducing this risk.

What To Think About

Bone marrow transplants are seldom used. Few children meet the criteria. And donors are almost always siblings, so finding a matching bone marrow donor may not be possible. The cost of the procedure is also a barrier.

About 1 out of 10 people with sickle cell disease have a matching donor.footnote 5

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